The Ladders to Cures Accelerator aims to develop treatments for multiple rare diseases through shared biological mechanisms.

Shionogi’s IntraBio acquisition brings Aqneursa, a drug approved for treating two rare neurological disorders and the potential to expand to additional indications, both rare and common. It’s Shionogi ...

One in 5 of the highest-revenue drugs of 2022 was exclusively approved for rare conditions, accounting for more than 7% of US pharmaceutical spending. Study Design: Cross-sectional study. Methods: ...

Stecker is a rare disease mother and advocate and served as assistant secretary of public affairs and deputy chief of staff at the Department of Health and Human Services. Every parent dreams of a ...

Researchers at the Greenwood Genetic Center (GGC) led the discovery of an unexpected connection between inherited blindness ...

Researchers from Kennedy Krieger Institute, University of Rochester Medicine and five other institutions released a new ...

The Cherokee Nation held its third annual Rare Disease Summit to advance policy, care, and early intervention initiatives for ...

A mixed-methods study of 149 rare disease patients and caregivers identifies centralized health data, telehealth, ...

This voice experience is generated by AI. Learn more. This voice experience is generated by AI. Learn more. Mahzi Therapeutics is pioneering a new biotech model by letting patients and families invest ...

An eight-year study of 3,496 rare disease hospitalizations at a tertiary hospital in northern Henan reveals rising admission ...